Child Friendly Medicines: Availability, Pharmaceutical design, Usability and patient outcomes

Abstract

Introduction Child and maternal health are key to overall human life expectancy, implying an urgent need for medicines that keep (unborn) children and mothers healthy and alive. Unfortunately, medicines may bring harm as well. For example, limb malformation due to the use of thalidomide by pregnant woman, or renal failure due to the use of paracetamol solutions or cough syrups that turned out to be contaminated with the toxic, but cheap solvens dietylenglycol. Availability A study in the Netherlands by the University of Utrecht, the University Medical Center Utrecht, the National Institute for Public Health and the Environment (RIVM) and the Medicines Evaluation Board (MEB) showed that only half of the commercially available and authorised medicines for use in adults were also available for use in children of any age between birth and 18 years. Moreover, the study indicated that authorised medicines were generally more scarce for younger than for older children and that some of the authorised medicines were not adequately addressing the needs of the child (e.g. inadequate dosing flexibility, too large tablet, too much ethanol per single dose). In addition, it turned out that some industries had not updated the medicines’ product information (SmPC) according to the current standards. As a result, it may not be fully clear to health care professionals and patients for which age group the medicine has been approved. The lack of authorised and well-designed i.e. age-appropriate or child friendly medicines is currently no longer considered ethical. Therefore, the US government, European Union and World Health Organization have implemented incentives to improve this situation. Although it is still too early to come to a final conclusion, the Dutch study indicated that the European Regulation is likely to reach its goal. Pharmaceutical design The study also indicated that fundamental knowledge on the pharmaceutical development of medicines for children is still scarce and fragmented. In order to obtain essential information, the acceptability of and preference among four different types of oral formulations was investigated in infants and preschool children between 1 and 4 years of age. The children and parents generally preferred a small 4-mm tablet (also referred to as mini-tablet) and syrup over a suspension or powder. Usability of paediatric medicines Although it was not recommended, some of the parents choose to administer the formulations with a smaller or larger portion of food or drink. Parents were more likely to do so when child acceptability was low. Child acceptability was generally increased when the formulations were administered with (a larger portion) of food or drink. Acknowledging that tablets are commonly broken to lower the dose or to ease swallowing, the study also evaluated the suitability of tablet splitters as an alternative to breaking tablets by hand. The results indicate that tablets are best broken by hand and that the dosing accuracy of tablet splitters highly varies per trademark. In fact, the application of the worst trademark could have resulted in 40% over- or underdosing. Moreover, a kitchen knife turned out to provide more accurate doses than 3 of the 6 tablet splitters investigated.